Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

by Alessandro Fraldi, Marta Serafini, Nicolina Cristina Sorrentino, Bernhard Gentner, Alessandro Aiuti, Maria Ester Bernardo
Published 2018-11-01

Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments.

by Carmen Vitiello, Stefania Faraso, Nicolina Cristina Sorrentino, Giovanni Di Salvo, Edoardo Nusco, Gerardo Nigro, Luisa Cutillo, Raffaele Calabrò, Alberto Auricchio, Vincenzo Nigro
Published 2009-01-01

Retinal Degeneration in MPS-IIIA Mouse Model

by Daniela Intartaglia, Giuliana Giamundo, Elena Marrocco, Veronica Maffia, Francesco Giuseppe Salierno, Edoardo Nusco, Alessandro Fraldi, Alessandro Fraldi, Ivan Conte, Ivan Conte, Nicolina Cristina Sorrentino
Published 2020-03-01

Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA

by Nicolina Cristina Sorrentino, Vincenzo Cacace, Maria De Risi, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Yan Huang, Nan Liu, Susan L. Kalled, Vivian W. Choi, Elvira De Leonibus, Alessandro Fraldi
Published 2019-12-01